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Our first review on progress and prospects in cystic fibrosis (CF) gene therapy was published in this series in October 2002. We now summarize the progress made since then and comment on the prospects for CF gene therapy over the next couple of years. Three clinical trials have been carried out, further supporting the proof-of-principle that gene transfer to the airway epithelium is feasible. Developments in viral and non-viral vectors, as well as recent alternative strategies such as gene repair, trans-splicing and stem cell therapy will be reviewed.

Original publication




Journal article


Gene Ther

Publication Date





1061 - 1067


Adenoviridae, Clinical Trials as Topic, Cystic Fibrosis, Dependovirus, Forecasting, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Respiratory Mucosa, Stem Cells